Patients And Biopharma: Trusted Partners In Fighting Rare Diseases

By Patrick McEnany, cofounder, chairman, president, and CEO, Catalyst Pharmaceuticals

Significant advances have been made in fighting rare diseases over the past decades. In the 10 years prior to the passage of the Orphan Drug Act in 1983, fewer than 10 drugs for rare diseases were approved by the U.S. FDA. Since then, an estimated 400 rare-disease therapies have been approved. In 2014, rare diseases comprised more than 40 percent of the 41 new drugs approved by the FDA — the highest number since the Orphan Drug Act was introduced. Just last year, 18 of the 46 new drugs approved by the FDA were for the treatment of rare diseases. The FDA continues to invest heavily in research of treatment for rare diseases, spending about $14 million annually on clinical trials through the Orphan Products Grants Program.